Orphan Drug Market to Worth USD 359.86 Billion by 2030 | Skyquest Technology

The Orphan Drug market is segmented based on disease and drug types. Based on the disease type, the global orphan drug market is segmented into oncological diseases, metabolic diseases, hematologic & immunologic diseases, infectious diseases, neurologic diseases, and other rare diseases and others. Based on drug type, the global orphan drug market is segmented as biologics and non-biologics- Industry Forecast 2023-2030


Westford USA, July 09, 2023 (GLOBE NEWSWIRE) -- According to SkyQuest, the rising prevalence of rare diseases is a significant driver contributing to the growth of the orphan drug market. Rare diseases, also known as orphan diseases, are medical conditions that affect a small percentage of the population. These diseases are characterized by their low prevalence, often occurring in less than 7 out of 10,000 individuals.

Browse in-depth TOC on the "Orphan Drug Market."

  • Pages - 211
  • Tables - 65
  • Figures – 75

The increasing prevalence of cancer and other rare genetic disorders primarily drives the growth of the orphan drug market. The rising incidence of these diseases has created a significant unmet medical need, prompting pharmaceutical manufacturers to develop innovative orphan drugs targeting specific genetic mutations or rare diseases. These drugs offer personalized therapy options to patients, addressing their particular medical conditions and improving treatment outcomes.

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Report Scope & Segmentation:

Attributes Details
Market Size in 2022 USD 174.11 Billion
2030 Value Projection USD 359.86 Billion
CAGR 9.5%
Forecast Period 2023-2030
Segments Covered
  • Disease Type
  • Drug Type
Regions Covered
  • North America
  • Europe
  • Asia Pacific
  • Latin America
  • Middle East and Africa

Prominent Players in the Orphan Drug Market

  • Vertex Pharmaceuticals
  • Alexion Pharmaceuticals
  • BioMarin Pharmaceutical
  • Shire (now part of Takeda)
  • Sanofi Genzyme
  • Celgene (now part of Bristol Myers Squibb)
  • Genentech (a subsidiary of Roche)
  • Pfizer
  • Novartis
  • Johnson & Johnson
  • AbbVie
  • Amgen
  • Ultragenyx Pharmaceutical
  • CSL Behring
  • Merck & Co.
  • Grifols
  • Sobi (Swedish Orphan Biovitrum)
  • Ipsen
  • Jazz Pharmaceuticals
  • AstraZeneca

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Oncology Segment is Expected to Grow in the Market Due to Rising High Prevalence of Cancer

The oncology segment emerged as the dominant sector in the orphan drug market. This dominance can be attributed to several key factors. The product development pipelines of key players in the pharmaceutical industry are heavily focused on oncology drugs. The high prevalence of cancer and the continuous need for innovative and effective therapies drive significant investment and research in this area.

The markets in North America are anticipated to experience rapid growth in the orphan drug market. This growth can be attributed to the increasing number of patients in the region susceptible to rare diseases. Unhealthy lifestyles and high-stress levels are key factors contributing to the spread of these diseases in this area.

Hematology Segment is Expected to Dominate the Market Due to the Increasing Number of Positive Regulatory Approvals

The hematology segment is expected to be the second most dominant segment in the orphan drugs market. This projection is based on several factors, including the continuous introduction of new products and the increasing number of positive regulatory approvals in the field of hematology.

Regional markets in Europe are poised for growth in the coming years, driven by factors such as the large patient population in the region and the increasing utilization of complex medications for rare diseases in the orphan drug market. Europe is household to many patients with rare diseases, creating a demand for specialized therapies that target these conditions.

A comprehensive analysis of the major players in the orphan drug market has been recently conducted in a report. The report encompasses various aspects, including collaborations, mergers, innovative business policies, and strategies, providing valuable insights into key trends and breakthroughs in the market. Furthermore, the report scrutinizes the market share of the top segments and presents a detailed geographic analysis. Lastly, the report highlights the major players in the industry and their endeavours to develop innovative solutions to cater to the growing demand.

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Key Developments in the Orphan Drug Market

  • Shanghai Henlius Biotech recently announced that the US Food and Drug Administration (FDA) had granted orphan drug designation to serplulimab, also known as Hansizhuang, for treating small-cell lung cancer. This designation recognizes the potential of serplulimab to address an unmet medical need for a rare and serious condition. Orphan drug status provides incentives and support for developing therapies targeting rare diseases.
  • ABIONYX Pharma recently reported that the USFDA had granted orphan drug designation to Bio-HDL CER-001 for treating lecithin-cholesterol acyltransferase deficiency, also known as LCAT deficiency. This designation highlights the significant medical benefit Bio-HDL CER-001 may offer patients with this rare inherited disorder. The orphan drug status provides regulatory and financial incentives to encourage the development of therapies for rare diseases, ultimately aiming to improve patient outcomes and quality of life.

Key Questions Answered in Orphan Drug Market Report

  • What specific growth drivers are projected to impact the market during the forecast period?
  • List the top companies in the market and explain how they have achieved their positions of influence.
  • In what ways do regional trends and patterns differ within the global market, and how these differences shape the market's future growth?

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